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斑马鱼在遗传性视网膜变性疾病建模与治疗发现中的作用

The Power of Zebrafish in Disease Modeling and Therapy Discovery for Inherited Retinal Degeneration.

作者信息

Xiao Huanhuan, Marshall Randi, Saxena Meera T, Zhang Liyun

机构信息

Department of Ophthalmology, Wilmer Eye Institute, Johns Hopkins University, Baltimore, MD, USA.

出版信息

Adv Exp Med Biol. 2025;1468:229-233. doi: 10.1007/978-3-031-76550-6_38.

Abstract

In the research of inherited retinal degeneration (IRD), zebrafish have emerged as a powerful model system, offering profound insights into disease mechanisms and opening new therapeutic avenues. This mini-review discusses the distinctive advantages that zebrafish provide for investigating retinal degeneration. It outlines contemporary genetic tools, with a specific focus on advanced CRISPR/Cas9 gene targeting technology, utilized for genome manipulation and disease modeling in zebrafish. By emphasizing the pivotal role of zebrafish in large-scale high-throughput drug discovery and the exploration of innovative gene therapy strategies, this succinct review underscores the adaptability and significance of the zebrafish model in advancing IRD research. It establishes a robust foundation for future studies and therapeutic developments in the field.

摘要

在遗传性视网膜变性(IRD)的研究中,斑马鱼已成为一个强大的模型系统,为疾病机制提供了深刻见解,并开辟了新的治疗途径。这篇综述讨论了斑马鱼在研究视网膜变性方面所具有的独特优势。它概述了当代遗传工具,特别关注用于斑马鱼基因组操作和疾病建模的先进CRISPR/Cas9基因靶向技术。通过强调斑马鱼在大规模高通量药物发现和创新基因治疗策略探索中的关键作用,这篇简洁的综述强调了斑马鱼模型在推进IRD研究中的适应性和重要性。它为该领域未来的研究和治疗发展奠定了坚实基础。

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